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Article

A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study

by
Monia Bengherbia
1,†,
Marc Berger
2,
Bénédicte Hivert
3,
Florian Rigaudier
4,
Luc Bracoud
5,
Ole Vaeterlein
6,
Karima Yousfi
1,
Michele Maric
7,
Marie Malcles
7 and
Nadia Belmatoug
1,*
1
Department of Internal Medicine, Referral Center for Lysosomal Diseases, Beaujon Hospital, AP-HP, Université Paris Cité, 92110 Clichy, France
2
Department of Biological and Clinical Hematology, Estaing Hospital, CHU Clermont-Ferrand, 63000 Clermont-Ferrand, France
3
Department of Hematology, Saint Vincent de Paul Hospital, GHICL, 59000 Lille, France
4
CEN Biotech, 21000 Dijon, France
5
Clario Inc. (Formerly Bioclinica, Inc.), 69006 Lyon, France
6
Clario Inc. (Formerly Bioclinica, Inc.), 20355 Hamburg, Germany
7
Takeda France SAS, 75116 Paris, France
*
Author to whom correspondence should be addressed.
†
Current address: Takeda France SAS, 75116 Paris, France.
J. Clin. Med. 2024, 13(10), 2926; https://doi.org/10.3390/jcm13102926
Submission received: 8 April 2024 / Revised: 10 May 2024 / Accepted: 11 May 2024 / Published: 16 May 2024
(This article belongs to the Section Clinical Research Methods)
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Abstract

Background/Objectives: Gaucher disease type 1 (GD1) is characterized by hepatosplenomegaly, thrombocytopenia, and disabling bone manifestations requiring regular MRI monitoring. The EIROS study assessed the real-world impact of velaglucerase alfa on GD1 bone disease, using MRI data collected in French clinical practice. Methods: MRIs collected retrospectively from treatment initiation and prospectively during follow-up (12-months) were analyzed centrally by a blinded expert radiologist to evaluate bone infiltration using the Bone Marrow Burden (BMB) score and a qualitative method (stable, improved or worsened for the spine and femur). Abdominal MRIs were also centrally analyzed to assess hepatosplenomegaly. Bone manifestations, hepatosplenomegaly, and hematologic parameters were analyzed from medical records. Results: MRI data were available for 20 patients: 6 treatment-naive patients and 14 patients who switched to velaglucerase alfa from another GD treatment. Interpretable MRIs for BMB scoring were available for seven patients for the spine and one patient for the femur. Qualitative assessments (n = 18) revealed stability in spine and femur infiltration in 100.0% and 84.6% of treatment-switched patients (n = 13), respectively, and improvements in 80.0% and 60.0% of treatment-naive patients (n = 5), respectively; no worsening of bone infiltration was observed. Liver, spleen, and hematologic parameters improved in treatment-naive patients and remained stable in treatment-switched patients. Conclusions: The qualitative real-world data support findings from clinical trials suggesting the long-term effectiveness of velaglucerase alfa on GD1 bone manifestations. When MRI assessment by radiologists with experience of GD is not possible, a simplified qualitative assessment may be sufficient in clinical practice for monitoring bone disease progression and treatment response.
Keywords: Gaucher disease; lysosomal storage disorder; bone marrow infiltration; hepatosplenomegaly; thrombocytopenia; real-world data; enzyme replacement therapy; velaglucerase alfa; magnetic resonance imaging Gaucher disease; lysosomal storage disorder; bone marrow infiltration; hepatosplenomegaly; thrombocytopenia; real-world data; enzyme replacement therapy; velaglucerase alfa; magnetic resonance imaging

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MDPI and ACS Style

Bengherbia, M.; Berger, M.; Hivert, B.; Rigaudier, F.; Bracoud, L.; Vaeterlein, O.; Yousfi, K.; Maric, M.; Malcles, M.; Belmatoug, N. A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study. J. Clin. Med. 2024, 13, 2926. https://doi.org/10.3390/jcm13102926

AMA Style

Bengherbia M, Berger M, Hivert B, Rigaudier F, Bracoud L, Vaeterlein O, Yousfi K, Maric M, Malcles M, Belmatoug N. A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study. Journal of Clinical Medicine. 2024; 13(10):2926. https://doi.org/10.3390/jcm13102926

Chicago/Turabian Style

Bengherbia, Monia, Marc Berger, Bénédicte Hivert, Florian Rigaudier, Luc Bracoud, Ole Vaeterlein, Karima Yousfi, Michele Maric, Marie Malcles, and Nadia Belmatoug. 2024. "A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study" Journal of Clinical Medicine 13, no. 10: 2926. https://doi.org/10.3390/jcm13102926

APA Style

Bengherbia, M., Berger, M., Hivert, B., Rigaudier, F., Bracoud, L., Vaeterlein, O., Yousfi, K., Maric, M., Malcles, M., & Belmatoug, N. (2024). A Real-World Investigation of MRI Changes in Bone in Patients with Type 1 Gaucher Disease Treated with Velaglucerase Alfa: The EIROS Study. Journal of Clinical Medicine, 13(10), 2926. https://doi.org/10.3390/jcm13102926

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